Effective treatment for rare leukemia

Dr. Fred Appelbaum, director of the Center's Clinical Research Division, helped conceive and conduct an NCI trial which found a significantly higher remission rate in acute promyelocytic leukemia patients treated with the chemotherapy drug arsenic trioxide.

Photo by Stephanie Cartier

Adult patients who received the chemotherapy drug arsenic trioxide (Trisenox) immediately after standard chemotherapy had a significantly higher remission rate and better overall survival than those who received standard chemotherapy, according results of a phase III cancer clinical trial for an uncommon form of leukemia.

Seventy-seven percent of adult patients with acute promyelocytic leukemia who received arsenic trioxide remained alive and in remission, free of relapse, three years after diagnosis. This compared with 59 percent on the standard treatment. The greater effectiveness of the experimental combination also resulted in better overall survival of 86 percent after three years for the patients who received the arsenic trioxide compared to 77 percent for patients on the standard treatment arm.

Dr. Fred Appelbaum, director of the Center's Clinical Research Division, helped conceive, write the protocol, enroll patients and write up the results for the NCI-sponsored trial.

Acute promyelocytic leukemia, a subtype of acute myeloid leukemia, accounts for approximately 10 percent of AML cases, or about 1,500 cases per year, in the United States. It is most often diagnosed in young and middle aged adults, but it also occurs in children and older adults.